Trial Outcomes & Findings for Vosaroxin and Infusional Cytarabine in Treating Patients With Untreated Acute Myeloid Leukemia (NCT NCT02658487)

Last Updated: 2025-04-15

Results Overview

CR is calculated as the percentage of patients with complete remission after the therapy. The response criteria is based on International Working Group Criteria. Complete Remission: Neutrophils(cells/μl)\>1000, Platelets (plt/μl)≥ 100,000,Bone marrow blasts (%) \<5; CR with incomplete count recovery (CRi): meets criteria for CR except for neutrophils ≤1000 or Meets criteria for CR except for platelets\< 100,000; Partial Remission: CR except for Bone marrow blasts (%) Decrease of ≥ 50% to value between 5% and 25%; Treatment Failure:Persistent acute myeloid leukemia in blood or bone marrow, or therapy fails to achieve a remission of any category, or death prior to response assessment

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

42 participants

Primary outcome timeframe

Up to 3 months

Results posted on

2025-04-15

Participant Flow

The recruitment period for this trial was March 2016 to April 2019. Participants were recruited at Vanderbilt University Medical Center, Yale University, and University Medical School, South Carolina.

42 participants met all eligibility criteria and were enrolled on this study. Nine of the participants received a second induction.

Participant milestones

Participant milestones
Measure	Treatment (Vosaroxin, Cytarabine) Induction Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Overall Study STARTED	42
Overall Study COMPLETED	26
Overall Study NOT COMPLETED	16

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Vosaroxin, Cytarabine) Induction Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Overall Study Death	2
Overall Study Disease progression	10
Overall Study Alternative therapy	1
Overall Study Adverse Event	1
Overall Study Treatment Failure, circulating blasts	1
Overall Study Refractory acute myeloid leukemia (AML)	1

Baseline Characteristics

Vosaroxin and Infusional Cytarabine in Treating Patients With Untreated Acute Myeloid Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Vosaroxin, Cytarabine) n=42 Participants Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	25 Participants n=5 Participants
Age, Categorical >=65 years	17 Participants n=5 Participants
Age, Continuous	64.4 Years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants
Race/Ethnicity, Customized White	35 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	5 Participants n=5 Participants
Race/Ethnicity, Customized Unknown/unreported	2 Participants n=5 Participants
Region of Enrollment United States	42 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 3 months

Population: Evaluable patients

Outcome measures

Outcome measures
Measure	Treatment (Vosaroxin, Cytarabine) n=42 Participants Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Complete Remission Rate (CR)	20 Participants

SECONDARY outcome

Timeframe: From start of therapy up to 1 year

Population: All patients participated

Survival distribution will be estimated using the method of Kaplan and Meier. Event-free survival time is defined as the time from start of therapy to progression or death for any reason (which ever comes first), and those alive without progression are censored at the last day of follow up.

Outcome measures

Outcome measures
Measure	Treatment (Vosaroxin, Cytarabine) n=42 Participants Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Event-free Survival	7.6 months Interval 2.9 to insufficient number of participants with events for estimating

SECONDARY outcome

Timeframe: Up to 3 months

Population: All participants

Events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. Counts of all events are summed up.

Outcome measures

Outcome measures
Measure	Treatment (Vosaroxin, Cytarabine) n=42 Participants Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Frequency of Grade 3-5 Adverse Event Related to Cytarabine and Vosaroxin (7+V)	133 adverse events

SECONDARY outcome

Timeframe: The time from complete remission to disease progression or death for any reason, assessed up to 1 year

Population: Patients who had complete remission after the therapy

Survival distribution will be estimated using the method of Kaplan and Meier. LFS time is defined as the time from complete remission to disease progression or death for any reason and those disease free and alive are censored at the last day of follow up.

Outcome measures

Outcome measures
Measure	Treatment (Vosaroxin, Cytarabine) n=20 Participants Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Leukemia-free Survival (LFS or DFS)	NA months Interval 13.1 to the median and upper bound are not estimable due to not long enough follow up

SECONDARY outcome

Timeframe: The time from start of therapy to death, assessed up to 1 year

Population: All participated patients

Survival distribution will be estimated using the method of Kaplan and Meier. The overall survival time is defined as time from start of therapy to death of any reason. Those alive are censored at the last day of known alive.

Outcome measures

Outcome measures
Measure	Treatment (Vosaroxin, Cytarabine) n=42 Participants Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I. Cytarabine: Given IV Vosaroxin: Given IV
Overall Survival	10.7 months Interval 7.4 to insufficient number of participants with events for estimating the upper bound

Adverse Events

Treatment (Vosaroxin, Cytarabine) Induction 1

Serious events: 10 serious events

Other events: 33 other events

Deaths: 18 deaths

Induction 2

Serious events: 5 serious events

Other events: 9 other events

Deaths: 4 deaths

Serious adverse events

Other adverse events

Additional Information

Stephen Strickland, MD

Vanderbilt University Medical Center

Phone: (615) 936-8422

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Treatment (Vosaroxin, Cytarabine) Induction 1 n=33 participants at risk Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Cytarabine: Given IV Vosaroxin: Given IV	Induction 2 n=9 participants at risk Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I.
Psychiatric disorders Altered mental state	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Renal and urinary disorders Hematuria	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Hepatobiliary disorders Cholecystitis	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Gastrointestinal disorders Acute sigmoid diverticulitis	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Electrocardiogram QT corrected interval prolonged	9.1% 3/33 • Number of events 4 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Gastrointestinal disorders Colitis	18.2% 6/33 • Number of events 8 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Renal and urinary disorders Acute kidney injury	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Blood and lymphatic system disorders Febrile Neutropenia	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Respiratory, thoracic and mediastinal disorders Respiratory failure	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Infections and infestations Sepsis	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Infections and infestations Fungemia	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Cardiac disorders Chronic heart failure	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Cardiac disorders Ventricular fibrillation	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Vascular disorders Thromboembolic event - Related to Hickman line placement	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Gastrointestinal disorders Typhlitis	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.

Measure	Treatment (Vosaroxin, Cytarabine) Induction 1 n=33 participants at risk Patients receive vosaroxin IV on days 1 and 4 and cytarabine IV continuously on days 1-7 (Induction I). Cytarabine: Given IV Vosaroxin: Given IV	Induction 2 n=9 participants at risk Patients with residual leukemia and for whom a second course is indicated in the judgment of the investigator may undergo a second course of treatment (Induction II) 14-57 days after day 1 of Induction I.
Blood and lymphatic system disorders Anemia	30.3% 10/33 • Number of events 10 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	22.2% 2/9 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Blood and lymphatic system disorders Febrile Neutropenia	81.8% 27/33 • Number of events 33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	55.6% 5/9 • Number of events 10 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Creatinine increased	6.1% 2/33 • Number of events 6 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Neutrophil count decreased	51.5% 17/33 • Number of events 36 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	44.4% 4/9 • Number of events 8 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Platelet count decreased	63.6% 21/33 • Number of events 55 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	55.6% 5/9 • Number of events 20 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Urine output decreased	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations White blood cell decreased	48.5% 16/33 • Number of events 29 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	55.6% 5/9 • Number of events 16 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Gastrointestinal disorders Colitis	15.2% 5/33 • Number of events 5 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Gastrointestinal disorders Diarrhea	78.8% 26/33 • Number of events 37 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	55.6% 5/9 • Number of events 19 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Gastrointestinal disorders Mucositis oral	33.3% 11/33 • Number of events 20 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	55.6% 5/9 • Number of events 9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hyperkalemia	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Anorexia	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hyperglycemia	15.2% 5/33 • Number of events 6 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hypokalemia	12.1% 4/33 • Number of events 5 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	22.2% 2/9 • Number of events 4 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hypoalbuminemia	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hypocalcemia	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hyponatremia	12.1% 4/33 • Number of events 5 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Metabolism and nutrition disorders Hypophosphatemia	9.1% 3/33 • Number of events 3 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Respiratory, thoracic and mediastinal disorders Dyspnea	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Respiratory, thoracic and mediastinal disorders Hypoxia	6.1% 2/33 • Number of events 3 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Psychiatric disorders Confusion	9.1% 3/33 • Number of events 3 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Infections and infestations Bacteremia/multiorgan failure	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Vascular disorders Catheter related thorombosis	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Infections and infestations Concern for fungal infection	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Neutropenic colitis	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Infections and infestations Staph bacteremia	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 4 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Blood and lymphatic system disorders Transaminitis	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Investigations Worsening Platelet Count Decrease	3.0% 1/33 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 1 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	6.1% 2/33 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	0.00% 0/9 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.
Infections and infestations Soft Tissue Infection	0.00% 0/33 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.	11.1% 1/9 • Number of events 2 • Adverse events that emerge (or worsen) after the first dose of study drug and within 30 days after the last dose.